DeCure for Autosomal dominant polycystic kidney disease
DeCure's autonomous Nephrology AI scientist is researching a drug-repurposing hypothesis for Autosomal dominant polycystic kidney disease — screening already-approved drugs against its 30-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleAutosomal dominant polycystic kidney disease maps to a 30-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.
Research record
01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash
Current lead
No approved-drug candidate for autosomal dominant polycystic kidney disease is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.
Molecular view
FKBP prolyl isomerase 1A (FKBP1A) — FKBP1A is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.
Loading structure…
helix sheet 4~{s},5~{r},6~{z},9~{s},10~{s},12~{e}drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6I1S · 1.52 Å · ligand (4~{S},5~{R},6~{Z},9~{S},10~{S},12~{E})-16-(ethylamino)-4,5-dimethyl-9,10,18-tris(oxidanyl)-3-oxabicyclo[12.4.0]octadeca-1(14),6,12,15,17-pentaene-2,8-dione (E26). Experimental structure, not a prediction.
What the evidence adds up to
A 2008 University of Maryland series reported on 20 patients who underwent simultaneous bilateral native nephrectomy and living donor renal transplantation for autosomal dominant polycystic kidney disease. Mean operative time for the nephrectomy portion alone was 190 minutes, with an average estimated blood loss of 723 cc. Mean hospital stay was 7.2 days. Graft survival was 100% and all patients reported relief of symptoms. The authors concluded the combined procedure could be done with minimal morbidity and that graft viability was not compromised.
A 1992 review noted that autosomal dominant polycystic kidney disease accounts for 7–10% of all patients on renal replacement therapy worldwide and was still regarded as untreatable, with poorly understood pathogenesis. A 2000 review described the disorder as among the most challenging human genetic disorders from both clinical and fundamental perspectives, but reported that progress had opened novel research avenues into genetic basis and cellular mechanisms. A 2016 review stated the disease is the most common inherited kidney disease in the United States, causing hypertension, cerebral aneurysms, nephrolithiasis, and kidney failure, and reviewed current treatment recommendations.
No drug therapy is mentioned in any of these abstracts. The 2008 surgical series is small, single-centre, and retrospective, with no control group. The reviews from 1992, 2000, and 2016 are narrative summaries that do not report any controlled trial data. What is still missing is any randomised evidence comparing simultaneous surgery to staged procedures, any drug therapy shown to slow disease progression in a controlled trial, and any patient stratification beyond the surgical candidacy criteria used in the Maryland series.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
The Journal of Urology · 2008 · 72 citations
Simultaneous Bilateral Native Nephrectomy and Living Donor Renal Transplantation are Successful for Polycystic Kidney Disease: The University of Maryland Experience
AbstractPURPOSE: Patients with autosomal dominant polycystic kidney disease have significant morbidity due to large kidney size and the resultant compression of adjacent organs. Surgical extirpation is limited to the most severe cases due to the risk of complications. Typically surgical extirpation of autosomal dominant polycystic kidney disease kidneys and renal transplantation are performed in staged fashion. The additive risks of these 2 procedures have been a barrier to a simultaneous surgical approach. The risks include transplant compromise due to cyst rupture, bleeding, adjacent organ injury and anti-HLA antibody sensitization from transfusion in cases of pretransplant nephrectomy. We reviewed the results of and graft survival data on bilateral nephrectomy for autosomal dominant polycystic kidney disease with simultaneous live donor renal transplantation. MATERIALS AND METHODS: From August 2003 to November 2007, 20 sets of kidneys were removed in patients with autosomal dominant polycystic kidney disease, followed by simultaneous live donor transplantation. We retrospectively reviewed the outcomes in terms of surgical time, complications, length of stay, transfusion rate and transplant kidney status. RESULTS: A total of 20 sets of kidneys were removed and these patients then underwent immediate live donor renal transplantation. Mean operative time was 190 minutes for the bilateral nephrectomy portion alone with an average estimated blood loss of 723 cc. Complications were rare and well tolerated. Mean hospital stay was 7.2 days for this procedure. Graft survival was 100% and all patients reported relief of symptoms. CONCLUSIONS: Bilateral nephrectomy and immediate transplantation in patients with autosomal dominant polycystic kidney disease can be done with minimal morbidity. Preliminary studies show that patients may have significant improvement in quality of life from this procedure and graft viability is not compromised.
Progress in molecular genetics of autosomal dominant polycystic kidney disease
AbstractAmong the prevalent human genetic disorders, human autosomal dominant polycystic kidney disease is certainly one of the most challenging, both from a clinical and a fundamental perspective. In the recent years, important progress opened novel research avenues to elucidate the genetic basis, the cellular pathophysiologic mechanisms and the molecular function of genes and proteins involved in autosomal dominant polycystic kidney disease.
Effect of processing on the structure and properties of oxide dispersion strenghthened (ODS) γ1 hardened superalloys
AbstractAutosomal dominant polycystic kidney disease is the most common inherited kidney disease and accounts for 7–10% of all patients on renal replacement therapy worldwide. Although first reported 500 years ago, this disorder is still regarded as untreatable and its pathogenesis is poorly understood despite much study. During the past 40 years, however, remarkable advances have transformed our understanding of how the disease develops and have led to rapid changes in diagnosis, prognosis, and treatment, especially during the past decade. This Review will summarise the key findings, highlight recent developments, and look ahead to the changes in clinical practice that will likely arise from the adoption of a new management framework for this major kidney disease.
Current recommendations for treating autosomal dominant polycystic kidney disease
AbstractAutosomal dominant polycystic kidney disease is the most common inherited kidney disease in the United States, causing hypertension, cerebral aneurysms, nephrolithiasis, and kidney failure. This article reviews the pathogenesis, diagnosis, associated complications, and current treatment recommendations for the condition.
Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), resolved on OpenAlex.
DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.
